GENE TRANSFER VECTOR HUMAN CLN2 CDNA BRAIN OF CHILDREN LINCL

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项研究的目的是治疗晚期婴儿神经元蜡样脂褐素增多症(LINCL)的中枢神经系统(CNS)表现，LINCL是一种致命的常染色体隐性遗传病。这将通过给大脑注射AAV2cuhCLN2来实现，AAV2cuhCLN2是一种血清2型腺相关病毒基因转移载体，表达在LINCL中发生突变的CLN2基因的正常编码序列。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The aim of this study is to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinoses (LINCL), a fatal, autosomal recessive disorder. This will be accomplished by administering to the brain AAV2cuhCLN2, a serotype 2 adenoassociated virus gene transfer vector expressing the normal coding sequences of the CLN2 gene, the gene that is mutated in LINCL.